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CRISPR/Cas9-mediated gene editing ameliorates neurotoxicity in mouse model of Huntington’s disease
Huntington’s disease is a neurodegenerative disorder caused by a polyglutamine repeat in the Huntingtin gene (HTT). Although suppressing the expression of mutant HTT (mHTT) has been explored as a therapeutic strategy to treat Huntington’s disease, considerable efforts have gone into developing allel...
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| Publicado no: | J Clin Invest |
|---|---|
| Main Authors: | , , , , , , , , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
American Society for Clinical Investigation
2017
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC5490741/ https://ncbi.nlm.nih.gov/pubmed/28628038 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1172/JCI92087 |
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